Background: Rapid advances in the understanding of the biology of MDS and AML have led to novel therapeutic interventions that have increased the clinical complexity of decision making in patient care. This study sought to quantify professional practice gaps and barriers to optimal care among healthcare providers treating patients with MDS and AML at academic medical centers and/or community cancer centers and clinics globally, with the goal of informing the design of evidence-based education interventions aiming at addressing these gaps.

Methods: In February-May 2021, 329 physicians (n = 190), physician assistants (n = 16) and advanced practice providers [nurse practitioners (n = 51) and pharmacists (n = 72)] were recruited to participate in a two-phase national educational study focused on MDS and AML. Eligible participants had to be active practitioners who treat patients with MDS and AML (n = 263 US; 66 ex-US). In the exploratory qualitative phase of this study, 30 US participants completed a 45-minute telephone interview focusing on the personal, contextual, and behavioral factors that influence a provider's clinical reasoning process in diagnosis and treatment. These participants completed a brief online case-based survey before the interview. Interviews were transcribed and analyzed through thematic analysis. The quantitative phase of this study consisted of an online survey composed of specific multiple-choice questions, semantic differential rating scales, and case vignettes. Respondents' answers to each of the questions in the quantitative survey were compared with optimal answers as identified by treatment guidelines and faculty experts.

Results: A group of 6 core practice gaps were identified through combined analysis of data from the online surveys and in-depth interviews. Of note, just 10% of US and 3% of ex-US participants agreed with expert opinion and would consider patients over 75 years with high-risk MDS or AML as possible candidates for stem cell transplant. For newly diagnosed high-risk MDS, 20% to 35% of participants are incorporating venetoclax/azacitidine into practice off-label, even for patients with TP53 mutations. About half (52%) of participants agreed with expert recommendation of venetoclax + HMA for elderly patients (>75 y) with newly diagnosed AML. A minority of participants' practice (18%) matched expert recommendations of venetoclax + HMA for a 68 yo patient with newly diagnosed AML and ECOG PS 2 with a FLT3 mutation with many participants (33%) recommending intensive chemotherapy with midostaurin. Similarly, for secondary AML progression from MDS with previous HMA therapy, a minority of participants (24%) agreed with the expert recommendation of CPX-351. Finally, a minority of study participants knew the mechanisms of action or targets of promising agents in clinical trials for patients with TP53 mutations, including eprenetapopt/APR-246 (31%) and magrolimab (36%). Detailed results of analysis will be presented, including group-specific analyses and investigation of the causalities of each of the practice gaps identified.

Conclusions: Many hematology/oncology professionals are not applying optimal care in patients with MDS and AML. Most notably, study participants may not adequately consider stem cell transplant for older patients with high-risk MDS or AML potentially impacting outcomes. At the same time, the overuse of intensive chemotherapy regimens by healthcare providers could have multiple consequences for older patients with AML including poorer outcomes and reduced quality of life. Finally, a lack of familiarity with promising agents under clinical investigation in MDS and AML with TP53 mutations may lead to missed opportunities to enroll eligible patients on clinical trials and potentially delay integration of emerging data and agents with new indications into clinical practice. Results of this study provide evidence to support the design of clinical tools, educational programs, and performance improvement interventions.


Daver:Bristol Myers Squibb: Consultancy, Research Funding; Genentech: Consultancy, Research Funding; Daiichi Sankyo: Consultancy, Research Funding; Glycomimetics: Research Funding; Gilead Sciences, Inc.: Consultancy, Research Funding; Sevier: Consultancy, Research Funding; Abbvie: Consultancy, Research Funding; Novimmune: Research Funding; FATE Therapeutics: Research Funding; Astellas: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; ImmunoGen: Consultancy, Research Funding; Trillium: Consultancy, Research Funding; Trovagene: Consultancy, Research Funding; Hanmi: Research Funding; Novartis: Consultancy; Jazz Pharmaceuticals: Consultancy, Other: Data Monitoring Committee member; Dava Oncology (Arog): Consultancy; Celgene: Consultancy; Syndax: Consultancy; Shattuck Labs: Consultancy; Agios: Consultancy; Kite Pharmaceuticals: Consultancy; SOBI: Consultancy; STAR Therapeutics: Consultancy; Karyopharm: Research Funding; Newave: Research Funding. Stein:Janssen Pharmaceuticals: Consultancy; Genentech: Consultancy; Astellas: Consultancy; Syndax Pharmaceuticals: Consultancy; Daiichi Sankyo: Consultancy; Syros Pharmaceuticals, Inc.: Consultancy; PinotBio: Consultancy; Celgene: Consultancy; Bristol Myers Squibb: Consultancy; Foghorn Therapeutics: Consultancy; Jazz Pharmaceuticals: Consultancy; Gilead Sciences, Inc.: Consultancy; Blueprint Medicines: Consultancy; Abbvie: Consultancy; Agios Pharmaceuticals, Inc: Consultancy; Novartis: Consultancy.

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